OBJECTIVES: To examine the current EU5 market access landscape for ATMPs

METHODS: Assessed the regulatory path, HTA and reimbursement for ATMPs with EC approval as of March 2018.

RESULTS: Ten ATMPs approved by the EC as of March 2018 of which one was suspended, two were withdrawn and one manufacturer did not renew marketing authorization. 6 ATMPs are currently available in Europe, with one approved recently (March 2018).

Market access status of available ATMPs varies considerably across the EU5:

• France: None of the available ATMPs are currently reimbursed. Only one evaluation completed by the HAS, with price negotiations still ongoing.
• Germany: 5 of the ATMPs are authorized by the Paul Ehrlich Institute, with three listed on the Lauer Taxe. Two were classified as procedures and therefore not assessed under AMNOG. Only one AMNOG evaluation completed so far with no additional benefit rating.
• Italy: Four ATMPs are currently authorized; three are reimbursed with financial managed entry agreements and P&R negotiations are ongoing for one ATMP.
• Spain: Only three ATMPs are currently authorized but none are marketed to date (P&R negotiations are ongoing).
• UK: Four ATMPs are currently available in the UK with positive NICE assessments based on a negotiated patient access scheme. Only one assessment conducted by NICE Highly Specialized Technologies (HST), all others evaluated under standard technology assessment (STA) pathway. None of the ATMPs have been assessed by the SMC.

CONCLUSIONS: Almost nine years since the first ATMP was approved, market access remains challenging, particularly in France and Spain. On the other hand, countries like Germany, Italy and the UK seem to be developing market access pathways primarily based on conditional reimbursement and outcomes/financial based agreements. Evolution of payment models and adaptation by countries will continue, as more ATMPs (e.g. CAR-T therapies) are launched, bringing important health benefits, but also substantially impacting pharmaceutical budgets.